In recent years, decisions made by the Food and Drug Administration have increasingly moved into the public eye. The agency oversees some of the most politically fraught treatments — abortion medication, opioids, Covid therapies and vaccines. Patients and caregivers are also more vocal about their desires, amplified by social media: You may have seen campaigns from families seeking F.D.A. approval for a drug that has a chance of treating their child’s rare disease.
Dr. Marty Makary, President Trump’s nominee to lead the F.D.A., would step into a role increasingly defined by the tension between fostering lifesaving innovation and ensuring that the public is protected from unsafe or ineffective drugs. This will be made even more complicated by the Trump administration’s threats to research funding and the distrust of science espoused by Dr. Makary’s boss, Robert F. Kennedy Jr.
The F.D.A. has long been maligned for being too slow in approving drugs for patients with fatal diseases. But when the agency approves a treatment with less robust data to prove its effectiveness, it is maligned for being influenced by the pharmaceutical industry or patient advocacy groups. The rise of gene therapies and other targeted treatments that promise to shift the course of rare diseases will only make this balancing act harder: These therapies can’t necessarily be tested in traditional rigorous, large-scale trials because of the inherently small number of people with the diagnosis.
Patients have long pushed the F.D.A. on its decisions. In the late 1980s, as the AIDS crisis devastated America, activists surrounded the F.D.A. headquarters to protest what they perceived as overly slow and even obstructionist drug approval processes. Patients were dying as the F.D.A. was waiting for better data to decide whether to approve a drug. Activists argued that there had to be a way to get promising treatments into the hands of patients, outside of clinical trials, before studies were complete.
This pressure led the F.D.A. in the early 1990s to develop what’s called the accelerated approval pathway, which allows drugs for serious, incurable diseases with promising data to be granted approval while larger-scale studies continue. The approval is conditional, meaning that if the treatment doesn’t end up showing a benefit in follow-up studies, the drug can be taken off the market. This was a major step forward for patients, granting them access to drugs that might not have been available within their lifetimes.
But this, too, is an imperfect process. Studies have questioned how many of these drugs ultimately show success. For instance, a recent review of cancer drugs that were granted accelerated approval revealed that fewer than half of them helped patients in follow-up trials. And those that didn’t were slow to be removed from the market.
This isn’t an argument against accelerated approval, but instead a caution. It is tempting to think: Why not let a patient try a drug if she is dying anyway? But approving ineffective drugs hurts the larger system. And it’s not what patients or their caregivers want — what they want is an agency that is flexible enough to give them the opportunity to try drugs that have the chance to be effective, even if that chance is small.
Research recognizes two types of error. The first is a Type 1 error, or “false positive,” which means approving a drug that doesn’t work or causes harm. There’s also a Type 2 error, or a “false negative,” where a drug that works is wrongly rejected. Both errors can cause harm. Deciding which risk to tolerate has long been the F.D.A.’s role, but rising external pressures are making that decision more complex.
That balance is also not necessarily the same for each disease, or even each point within the disease process. Someone early on in a disease, even a fatal one, may be less willing to tolerate harsh side effects for limited benefit. Researchers have proposed mathematical models for this sort of patient-centered calculus that could play into F.D.A. decision-making, but as of now this is not taken into account in a structured way.
How will Dr. Makary reconcile all the different priorities? As a researcher who has offered a vocal critique of our medical system and argued for greater transparency between doctors and patients, he could bring pragmatism to the post and lead with the science. Dr. Makary’s latest book, “Blind Spots,” examines how to separate medical dogma from evidence. Being willing to question authority while also critically assessing the quality of scientific research is what the F.D.A. needs right now, particularly to counterbalance Mr. Kennedy.
Leading the agency is about managing the increasing uncertainty in medicine, a weighty but necessary burden. Even if Dr. Makary is up to that task, time will tell whether thoughtful change is ultimately possible in our political climate. But when it is done well, we all benefit.
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